Paroxysmal Nocturnal Haemoglobinuria (PNH) Dr Conal McConville ...

ESA=erythropoietin stimulating agents
Historical Management of PNH
Palliative options do not impact progression and risk
for severe morbidities and mortality 1
• Transfusions
– Risk of iron overload
– Transient treatment of anaemia
• Anticoagulants
– Risk of haemorrhage
– Ineffective in many patients 2
• Red cell supplements
– ESAs may expand clones and elevate haemolysis
– Folic acid, iron, erythropoiesis-stimulating stimulating agents
• Steroids/androgen hormones
– No controlled clinical trials
– AE’s
1. International PNH Interest Group. Blood. 2005;106:3699-3709. 2. Hillmen P et al. Blood. 2007;110:4123-8.
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